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NCT05918211PHASE3Recruiting

Fontan Udenafil Exercise Longitudinal Assessment Trial - 2

Mezzion Pharma Co. Ltd

Start Date

10/30/2023

Completion Date

10/28/2025

Summary

This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure.

Detailed Description

This study is a 26 week, prospective, multicenter, randomized, double-blinded, placebo-controlled safety and efficacy study of udenafil vs. placebo in adolescent subjects who have had the Fontan procedure. The primary efficacy endpoint will be change from baseline at 26 weeks in peak minute oxygen consumption (VO2 mL/kg/min) as measured by maximal cardiopulmonary exercise test (CPET) reading laboratory who will be blinded to treatment allocation.

Eligibility Criteria

Age Range: 12 years to 18 years

Inclusion Criteria: 1. Males and females with Fontan physiology who are 12 to less than 19 years of age at enrollment. 2. Participant consent or parental/guardian consent and participant assent. 3. Participant fluency in primary language of country in which study is being conducted. 4. Current antiplatelet or anticoagulant therapy. Exclusion Criteria: 1. Height \< 132 cm. 2. Weight \< 40 kg. 3. Hospitalization for acute decompensated heart failure within the last 12 months. 4. Current intravenous inotropic drugs. 5. Undergoing evaluation for heart transplantation or listed for transplantation. 6. Diagnosis of active protein losing enteropathy or plastic bronchitis within the last 3 years, or a history of liver cirrhosis. 7. Known Fontan baffle obstruction, branch pulmonary artery stenosis, or pulmonary vein stenosis resulting in a mean gradient of \> 4 mmHg between the regions proximal and distal to the obstruction as measured by either catheterization or echocardiography, obtained prior to screening for the trial. 8. Single lung physiology with greater than 80% flow to one lung. 9. Failure to achieve maximal exertion (defined as RER \< 1.10) on screening/baseline exercise test. 10. Peak minute oxygen consumption (VO2) less than 45% or ≥ 80% of predicated for age and gender at enrollment. 11. Severe ventricular dysfunction assessed qualitatively by clinical echocardiography within 6 months prior to enrollment. 12. Severe valvar regurgitation, ventricular outflow obstruction, or severe aortic arch obstruction assessed by clinical echocardiography within six months prior to enrollment. 13. History of significant renal (serum creatinine \> 2.0), hepatic (serum AST and/or ALT \> 3 times upper limit of normal), gastrointestinal or biliary disorders that could impair absorption, metabolism or excretion of orally administered medications. 14. Inability to complete exercise testing at baseline screening. 15. Subjects with a pacemaker whose heart rate at peak exercise is controlled by the extrinsic pacemaker as opposed to a native atrial rhythm. 16. History of PDE-5 inhibitor use within 12 months prior to enrollment. (Treatment is defined as chronic therapy as opposed to a single dose.) 17. History of any other medication for treatment of pulmonary hypertension within 3 months before study onset. 18. Known intolerance to oral udenafil. 19. Frequent use of medications or other substances that inhibit or induce CYP3A4. 20. Current use of alpha-blockers or nitrates. 21. Ongoing or planned participation in another research protocol that would either prevent successful completion of planned study testing or invalidate its results. 22. Noncardiac medical, psychiatric, and/or social disorder that would prevent successful completion of planned study testing or would invalidate its results. 23. Cardiac care, ongoing or planned, at a non-study center that would impede study completion. 24. For females: Pregnancy at the time of screening, pregnancy planned before study completion, or refusal to use an acceptable method of contraception for study duration if sexually active. 25. Unable to abstain or limit intake of grapefruit juice and grapefruit containing drinks during the duration of the trial. 26. Refusal to provide written informed consent/assent. 27. In the opinion of the investigator, the subject is likely to be non-compliant with the study protocol. 28. History of clinically significant thromboembolic event, in the option of the site Principal Investigator, that may put the subject at increased risk of a subsequent event while participating in the study. 29. Coronavirus disease 2019 (COVID-19) vaccination or symptoms of COVID-19 infection within 7 days of Visit 1. 30. Not taking antiplatelet or anticoagulant therapy.

Interventions

DRUG

Udenafil

DRUG

Placebo

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Conditions

Single Ventricle Heart Disease

Locations

Phoenix Children's Hospital

Phoenix, Arizona 85016

United States

Arkansas Children's

Little Rock, Arkansas 72202

United States

Children's Hospital of Los Angeles

Los Angeles, California 90027

United States

Rady Children's Hospital

San Diego, California 92123

United States

UCSF Benioff Children's Hospital

San Francisco, California 94158

United States

Children's Hospital of Colorado

Denver, Colorado 80045

United States

Yale School of Medicine

New Haven, Connecticut 06510

United States

Nemours Children's Hospital

Wilmington, Delaware 19803

United States

Childrens National Medical Center

Washington D.C., District of Columbia 20010

United States

UF Health Shands Hospital

Gainesville, Florida 32608

United States

Joe DiMaggio Children's Hospital

Hollywood, Florida 33021

United States

Johns Hopkins All Children's Hospital

St. Petersburg, Florida 33701

United States

Children's Healthcare of Atlanta

Atlanta, Georgia 30329

United States

Children's Hospital of Georgia

Augusta, Georgia 30912

United States

Lurie Children's Hospital

Chicago, Illinois 60611

United States

University of Chicago

Chicago, Illinois 60637

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

University of Michigan

Ann Arbor, Michigan 48109

United States

Children's Mercy Hospital Kansas City

Kansas City, Missouri 64108

United States

Washington University

St Louis, Missouri 63130

United States

University of Nebraska Children's Hospital and Medical Center

Omaha, Nebraska 68114

United States

Mt. Sinai Children's Hospital

New York, New York 10029

United States

New York-Presbyterian Children's Hospital

New York, New York 10032

United States

Dana.Amaro@atriumhealth.org

Charlotte, North Carolina 28204

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio 45206

United States

Cleveland Clinic

Cleveland, Ohio 44195

United States

Nationwide Children's Hospital

Columbus, Ohio 43215

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania 15224

United States

MUSC Pediatric Research Group

Charleston, South Carolina 29425

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

UT Southwestern Medical Center

Dallas, Texas 75235

United States

Texas Children's Hospital

Houston, Texas 77030

United States

Primary Children's Medical Center

Salt Lake City, Utah 84113

United States

Seattle Children's Hospital

Seattle, Washington 98105

United States

Children's Hospital of Wisconsin

Milwaukee, Wisconsin 53226

United States

Sejong General Hospital

Bucheon-si, Gyeonggi-do 14754

South Korea

Seoul National University Children's Hospital

Seoul, 03080

South Korea

Yonsei Severance Hospital

Seoul, 03722

South Korea